Safety and Effectiveness of PCSK9 Inhibitors and Inclisiran in Patients With Neuromuscular Disorders and Statin Intolerance.

BACKGROUND AND

OBJECTIVES

Limited data exist on the efficacy and safety of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors and inclisiran among patients with neuromuscular disorders and statin-induced myotoxicity and/or hepatotoxicity. We assessed the safety and efficacy of PCSK9 inhibitors and inclisiran in this specific patient subgroup.

METHODS

We conducted an observational cohort study evaluating patients with available clinical and laboratory data prior to and at prespecified time points following treatment initiation with PCSK9 inhibitors or inclisiran.

RESULTS

Eleven patients with neuromuscular disorders and statin intolerance were included in this study. Median follow-up time after PCSK9 inhibitor or inclisiran initiation was 14 (9-17) months. PCSK9 inhibitors or inclisiran use led to a significant decrease in mean low-density lipoprotein cholesterol levels. Moreover, all patients tolerated these lipid-lowering agents without exacerbation of their underlying myositis, myopathy, neuromuscular junction disorder, and without presenting any adverse event or relapse of myotoxicity and/or hepatotoxicity.

DISCUSSION: The present real-world study highlights the potential therapeutic value of PCSK9 inhibitors and inclisiran among patients with neuromuscular disorders and statin intolerance. The findings of this study are in accordance with previous reports, showing that PCSK9 inhibitors are safe as a lipid lowering treatment for long-term use among patients with statin-associated immune-mediated necrotizing myopathy (IMNM).